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Your search for “RNA Therapeutics” returned 114 results

Simplifying RNA Editing for Treating Genetic Diseases

February 10, 2022

New research led by bioengineers at the University of California San Diego could make it much simpler to repair disease-causing mutations in RNA without compromising precision or efficiency. The new RNA editing technology holds promise as a gene therapy for treating genetic diseases.

Chemical Disguise Transforms RNAi Drug Delivery

November 17, 2014

Researchers at University of California, San Diego School of Medicine have developed a way to chemically disguise RNAi drugs so that they are able to enter cells. Once inside, cellular machinery converts these disguised drug precursors — called siRNNs — into active RNAi drugs.

For First Time, Scientists Use CRISPR-Cas9 to Target RNA in Live Cells

March 17, 2016

…efficient method for targeting RNA— intermediary genetic material that carries the genetic code from the cell’s nucleus to protein-making machinery — in living cells. Researchers at University of California, San Diego School of Medicine have now achieved this by applying the popular DNA-editing technique CRISPR-Cas9 to RNA. The study is…

Closing the RNA Loop Holds Promise for More Stable, Effective RNA Therapies

August 26, 2024

New methods to shape RNA molecules into circles could lead to more effective and long-lasting therapies, shows a study by UC San Diego researchers. The advance holds promise for a range of diseases, offering a more enduring alternative to existing RNA therapies, which often suffer from short-lived effectiveness in the…

New Therapeutic Target Identified for ALS and Frontotemporal Degeneration

November 8, 2013

…Ludwig Institute for Cancer Research have identified a novel therapeutic approach for the most frequent genetic cause of ALS, a disorder of the regions of the brain and spinal cord that control voluntary muscle movement, and frontotemporal degeneration, the second most frequent dementia.

Add human-genome produced RNA to the list of cell surface molecules

September 10, 2020

Bioengineers at UC San Diego have shown that human-genome produced RNA is present on the surface of human cells, suggesting a more expanded role for RNA in cell-to-cell and cell-to-environment interactions than previously thought.

New Version of DNA Editing System Corrects Underlying Defects in RNA-based Diseases

August 10, 2017

…the technique to track RNA in live cells in a method called RNA-targeting Cas9. In a study published August 10 in Cell, the team took RCas9 a step further: they corrected molecular mistakes that lead to microsatellite repeat expansion diseases, which include a type of ALS and Huntington’s disease.

RNA-Based Drugs Give More Control Over Gene Editing

November 16, 2015

…Isis Pharmaceuticals demonstrate a commercially feasible way to use RNA to turn the CRISPR-Cas9 system on and off as desired — permanently editing a gene, but only temporarily activating CRISPR-Cas9.

Potential Drug Target for Difficult-To-Treat Breast Cancer: RNA-Binding Proteins

July 2, 2021

UC San Diego studies using human cell lines and tumors grown in mice provide early evidence that inhibiting RNA-binding proteins, a previously overlooked family of molecules, might provide a new approach for treating some cancers.

An Errant Editing Enzyme Promotes Tumor Suppressor Loss and Leukemia Propagation

January 3, 2019

UC San Diego researchers have found a stem cell enzyme copy edits more than 20 tumor types, providing new therapeutic target for preventing cancer cell resistance to chemotherapy and radiation.

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